PT  - JOURNAL ARTICLE
AU  - Vinall, Maria
TI  - Sebelipase Alfa Normalizes ALT and Multiple other Disease-Related Abnormalities in Patients with LAL-D
DP  - 2014  01
TA  - MD Conference Express
PG  - 14--15
VI  - 14
IP  - 48
4099  - http://mdc.sagepub.com/content/14/48/14.short
4100  - http://mdc.sagepub.com/content/14/48/14.full
AB  - Lysosomal acid lipase deficiency (LAL-D) is an autosomal genetic recessive disorder that leads to an inability to break down lipid particles in the lysosome, and it is associated with early-onset cirrhosis and cardiovascular disease. This article discusses the Acid Lipase Replacement Investigating Safety and Efficacy trial [ARISE; NCT01757184] was a randomized, double-blind, placebo-controlled phase 3 study designed to evaluate sebelipase alfa in patients with LAL-D.